FUJIFILM Corporation Announces Results from Phase II Clinical Trial of "T-817MA" in Patients with Alzheimer's Disease in the United States

FUJIFILM Corporation (TOKYO: 4901) (President: Kenji Sukeno) announced
that the company’s Alzheimer’s Disease drug “T-817MA,” did not meet its
primary study endpoints of cognition or global clinical function in its
Phase II clinical trial conducted in the United States on patients with
mild to moderate Alzheimer’s Disease (AD). There were no significant
differences in secondary outcomes. In exploratory analyses, change of
the cerebrospinal fluid (CSF) biomarker phospho-tau (p-Tau) benefited
from the higher dose treatment while hippocampal*1 volumes
decreased less in the lower doses group with statistical significance.
Post hoc analyses also suggested T-817MA treatment with shorter duration
of illness and symptoms was associated with better cognitive outcomes
with statistical significance. Fujifilm will review the results of the
Phase II clinical trial with regulatory authorities including FDA (U.S.
Food and Drug Administration) and will take necessary steps toward
further development including Phase III clinical trial of this compound.

A pre-clinical research, led by Dr. Rudolph Tanzi, Joseph P. and Rose F.
Kennedy Professor of Neurology at Harvard Medical School, and Vice-Chair
of Neurology at Massachusetts General Hospital, revealed that T-817MA
acts on microglia*2 that have a risk gene for AD and promotes
the clearance of amyloid-β (Aβ).
These results suggest that T-817MA
may act on p-Tau and Aβ, major causal substances in AD.

AD’s main symptoms are abnormalities in cognitive functions, including
memory. It is believed that abnormality of neurological functions in AD
manifests as a result of Aβ protein deposits (senile plaques) in the
brain. The progression of the disease has strong association with the
formation of abnormal assemblies caused by hyperphosphorylation and
fibrillization of tau proteins, found in the axons of nerve cells
(neurofibrillary tangle), synapse*3 loss and atrophy of the
brain including hippocampus.
The number of people suffering from
dementia is estimated to be 44 million around the world. The number is
projected to increase to 76 million by 2030 with the aging of the world
population. Over half of these dementia cases suffer from AD, and the
trend is set to continue.
AD drugs currently available on the
market include acetylcholinesterase inhibitors, such as donepezil
hydrochloride. As these drugs address the deficit in neurotransmission
to give temporary improvement to the patients' symptoms, there is an
unmet need for a new class of therapies to treat AD.

T-817MA is an AD drug discovered by the Fujifilm Group company, Toyama
Chemical. It has potent neuroprotective effect and promotes neurite
outgrowth through the activation of sigma receptors*4,
demonstrating strong efficacy in animal models. Fujifilm is committed to
further accelerating the development of T-817MA under possible
partnerships, and contributing to resolving social challenges through
the delivery of innovative pharmaceutical products.

*1

Hippocampus is a region of the cerebrum, associated with memory. AD
causes neuronal loss, which results in hippocampal atrophy.

*2

Microglia are a type of glia cells, which are component of the
central nervous system. Microglia provide immune defense in the
brain, while also being involved in repairing abnormal neural cells,
and eliminating the aggregated Aβ. At the same time, excessively
activated microglia are known to play a role in neuro-degeneration
in the central nervous system.

*3

Synapse is a structure connecting neurons, allowing them to pass
electrical signals to convey information between them.

*4

Sigma receptors are protein that exists on the membrane of
endoplasmic reticulum in cells, and have the function of mitigating
neuro-degeneration in the central nervous system.